Cylene advances preclinical development of CX-5461 for treatment of cancer

Cylene Pharmaceuticals, Inc. has advanced its targeted, non-genotoxic, activator of p53 for the treatment of leukemias, lymphomas and multiple myeloma through formal preclinical development, the Company announced today.  The small molecule, CX-5461, avoids the direct DNA damaging side-effects typically seen with current p53 activating drugs by selectively inhibiting the newly-validated RNA Polymerase I (Pol I) cancer target. Upon activation the p53 protein functions as a tumor suppressor by causing cancerous cells to self-destruct.  By targeting Pol I to activate p53 in blood cancers, Cylene's first-in-class agent safely triggers the body's natural cancer fighting capability to selectively kill tumor cells.

The US Patent Office recently awarded patent coverage for CX-5461 to Cylene, thereby expanding the Company's extensive Pol I intellectual property portfolio. Cylene's unique mechanistic knowledge and chemistry expertise enabled creation of molecules that act through direct inhibition of Pol I, leading to disruption of the Mdm2-p53 interaction and activating p53 to kill cancer cells. The appealing mechanism of action, highly favorable preclinical profile, robust in vivo efficacy in multiple models and bioavailability in multiple species, underpin Cylene's decision to develop CX-5461 as a clinical candidate.

"CX-5461 looks like a promising agent for patients with leukemia, lymphoma and some types of solid tumors. Selective and potent activation of p53 by agents that do not damage DNA is a unique approach," stated Daniel D. Von Hoff, M.D., Cylene's Senior Vice President, Medical Affairs. "The successful targeting of Pol I provides the ideal non-genotoxic path for p53 activation and has the potential to deliver a new class of powerful anticancer therapies to our patients."

"Advancing CX-5461 toward the clinic is a real testament to our ability to capitalize on new targets and we take great pride in the fact that Cylene is leading the industry in exploiting p53 through this novel mechanism," commented William G. Rice, PhD, President and CEO of Cylene Pharmaceuticals. "We expect this molecule to satisfy a substantial need in the battle against leukemias, lymphoma and myelomas."